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Releasing brakes: Potential new methods for Duchenne muscular dystrophy therapies

Releasing brakes: Potential new methods for Duchenne muscular dystrophy therapies

Releasing brakes: Potential new methods for Duchenne muscular dystrophy therapies
Feb 24, 2020 1 min, 16 secs

The molecules tested by the team from the Perelman School of Medicine at the University of Pennsylvania eased repression of a specific gene, utrophin, in mouse muscle cells, allowing the body to produce more utrophin protein, which can be subbed in for dystrophin, a protein whose absence causes DMD.

Since the patient's body never produced dystrophin, it interprets the new micro-dystrophin protein as a foreign, hostile invader and attacks, which may lead to adverse events and nullify any benefits.

"We're using an approach that attempts to increase utrophin levels in the body because it has functional characteristics and a genetic structure similar to dystrophin.

Since the body already produces it, the immune system recognizes the protein as the body's own and does not attack it or the cells producing it, even when over-expressed," Khurana said.

There have been other attempts to use utrophin as a substitute for dystrophin using drugs, but those methods have focused on boosting utrophin through activating the "promoter," the part of a gene that kick-starts the process of its expression in a person.

Through this, they found 27 promising "hits." After ranking their effectiveness using an algorithm they developed called Hit to Lead Prioritization Score (H2LPS), 10 molecules were extensively tested in muscle cell lines, and the top-scoring molecule, trichostatin A (TSA), was tested in a mouse model of muscular dystrophy where it led to significant improvements in muscle structure and function.

With the molecules they identified, Khurana and his team believe they've found potential ways of developing therapies to treat DMD patients.

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